(Learn how and when to remove this template message)
Nasdaq: RARE
Life Sciences
Areas served
Key people
Emil Kakkis
president & founder
ProductsResearch Antibodies, Biotech
RevenueIncrease $271 Million(2020)[1]
Number of employees
740 (2019)

Ultragenyx is a biopharmaceutical company involved in the R&D of novel products for treatment of rare and ultra-rare genetic diseases such as Angelman syndrome and X-linked hypophosphatemia. The company has survived the failure of several products [2] and now collaborates with previous rivals Genetex, Kyowa Hakko Kirin, Mereo Biopharma and Daiichi Sankyo.[3] The products Burosumab, Triheptanoin and Vestronidase alfa have received FDA approval.


Ultragenyx Pharmaceutical Inc. was founded in 2010 by Emil Kakkis because of his interest in therapies for rare genetic diseases. He became the Chief executive officer and President and focused the company to "meeting the need for treatments for ultra-rare diseases" — understood as affecting fewer than one patient per 50,000 people.[4][5] In 2014 the USA-based company went public with an IPO that raised $126 million.[6] At that time it had four treatments in clinical trials.

Notable products

  • Burosumab (KRN-23; brandname Crysvita) has been approved as a therapy for X-linked hypophosphatemia.[7][8] It works by raising the levels of phosphate in the blood thereby making it possible for faster healing of bone lesions in osteomalacia.[9]
  • Triheptanoin, a purified medium-chain triglyceride, has been approved for the treatment of long-chain fatty acid oxidation disorders, in which the body is unable to produce energy from fat.[10] Due to its odd-chain properties, Triheptanoin is broken down into metabolites that replace deficient intermediates in the Citric Acid Cycle.[11]
  • Vestronidase alfa was approved in the United States in November 2017 to treat children and adults with the inherited metabolic condition mucopolysaccharidosis type VII (MPS VII), also known as Sly syndrome.[12]

Investigational products

Collaboration with Arcturus

Since 2015 Ultragenix has been collaborating with Arcturus Therapeutics to develop mRNA products. Arcturus' clinical programs also include a potential covid-19 vaccine candidate.[17]

Gene therapy technology

Ultragenyx acquired Dimension Therapeutics in 2017[19] to obtain adeno-associated virus (AAV)-based gene therapy manufacturing technology.[20]

Product failures

The company's recent research and development collaborations with competitors has followed failures with several in-house products. The company has been accused of making rushed and incomplete regulatory applications.[21] Investment banks Evercore and Jefferies noted that several potential products have "meaningful and underappreciated clinical risk."[22] [23]

  • Ace-ER's - Possible treatment for GNE myopathy was discontinued after failing to meet three key secondary endpoints that evaluated patients' physical functions, leg muscle strength and knee extension force.[22] Previous trials in mice had shown efficacy. [24]
  • UX007 - Stage 3 trials continued despite failing a mid stage study.[22]


  1. ^ "Ultragenyx Pharmaceutical Revenue 2012-2021 | RARE".
  2. ^ "Ultragenyx looks ahead after triheptanoin failure". March 23, 2017.
  3. ^ Denworth, Lydia (2020). "Is gene therapy ready to treat some forms of autism?". Science. doi:10.1126/science.abf2497.
  4. ^ "US National Institute of Health Emil Kakkis Interview". 2018.
  5. ^ "UCLA Medicine Fall 2001 Neufeld Steps Down as Department Chair with Big Plans for Her Future" (PDF). 2001.
  6. ^ "Ultragenyx raises $126 million in IPO". February 5, 2014.
  7. ^ Lamb, Y. N. (2018). "Burosumab: First Global Approval". Drugs. 78 (6): 707–714. doi:10.1007/s40265-018-0905-7. PMID 29679282. S2CID 5022649.
  8. ^ "Specialty Pipeline Monthly Update" (PDF). September 2018.
  9. ^ "FDA.gov". June 18, 2020.
  10. ^ "2020: Truly the Year Of the Specialty Drug". December 15, 2020.
  11. ^ "Baylor College of Medicine and Texas Children's Hospital". Retrieved December 27, 2020.
  12. ^ "Drug Trial Snapshot: Mepsevii". U.S. Food and Drug Administration (FDA). 4 December 2017. Archived from the original on 10 December 2019. Retrieved 9 December 2019. Public Domain This article incorporates text from this source, which is in the public domain.
  13. ^ "A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome (KIK-AS)". 2020. {{cite journal}}: Cite journal requires |journal= (help)
  14. ^ "GeneTx and Ultragenyx Announce Investigational New Drug (IND)". January 15, 2020.
  15. ^ Carpenter, T. O.; Whyte, M. P.; Imel, E. A.; Boot, A. M.; Högler, W.; Linglart, A.; Padidela, R.; Van't Hoff, W.; Mao, M.; Chen, C. Y.; Skrinar, A.; Kakkis, E.; San Martin, J.; Portale, A. A. (2018). "Burosumab Therapy in Children with X-Linked Hypophosphatemia". The New England Journal of Medicine. 378 (21): 1987–1998. doi:10.1056/NEJMoa1714641. hdl:1805/18603. PMID 29791829. S2CID 44135503.
  16. ^ "Mereo BioPharma Group Pact With Ultragenyx Pharmaceutical to Develop Setrusumab to Strengthen Bones". December 21, 2020.
  17. ^ "Global Legal Chronicle". June 1, 2020.
  18. ^ "mRNA therapeutics - Arcturus Therapeutics/Ultragenyx Pharmaceutical". Adis Insights, Springer. 21 Jun 2019.
  19. ^ "Bruised Ultragenyx attempts swoop for in-demand Dimension". September 18, 2017.
  20. ^ "Daiichi Sankyo Enters Partnership with Ultragenyx for a Total of $225 Million". March 31, 2020.
  21. ^ Geigert, John The Challenge of CMC Regulatory Compliance for Biopharmaceuticals, p. 420, at Google Books
  22. ^ a b c "Ultragenyx shelves genetic disease drug after trial failure". August 23, 2017.
  23. ^ Dayen, David Monopolized: Life in the Age of Corporate Power, p. 120, at Google Books
  24. ^ Takeda. Translational Research in Muscular Dystrophy, p. 72, at Google Books